Adeno-Associated Vectors for Gene Delivery to the Nervous System

Gene therapy approaches based on adeno-associated viral vector (AAV) systems offer many unique advantages for nervous system applications. These vectors are opening up new approaches for the treatment of neurodegenerative diseases. AAV can efficiently deliver genes to postmitotic neuronal cell types offering long-term expression, can be generated in high titers, and are associated with only minimal immunological complications. Numerous animal studies have demonstrated the efficiency of these vectors both at preclinical and clinical development stages. The current chapter will describe the basic features of AAV vectors, list few examples of their applications as a therapeutic tool to treat diseases of the central nervous system, and discuss progress in the manufacturing process.
Source: Springer protocols feed by Neuroscience - Category: Neuroscience Source Type: news